Plozasiran

PASADENA, Calif.--( BUSINESS WIRE )--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it is scheduled to participate in the following upcoming events: Piper Sandler 36th Annual Healthcare Conference – December 3-5, 2024 Type: Fireside Chat Presentation Date/Time: December 3, 2024, 1:30 p.m. EST 7 th Annual Evercore ISI HealthCONx Conference – December 3-5, 2024 Type: Fireside Chat Presentation Date/Time: December 4, 2024, 2:10 p.m. EST The APA/JPS/CAP/IAP 2024 Joint Meeting – December 9-12, 2024 Title: PALISADE: A Phase 3 Study to Assess the Efficacy and Safety of Plozasiran in adults with Genetically or Clinically-Defined Familial Chylomicronemia Syndrome (FCS) at high risk of Acute Pancreatitis (AP) Date/Time: December 9, 2024, 12:00 p.m. HST Presenter: Eleonora Goldberg Session: Poster RNA at the Bench and Bedside IV – December 9-11, 2024 Title: Developing siRNA for Neurodegenerative Disorders Date/Time: December 9, 2024, 2:10 p.m. PST Presenter: Christine Esau Session: Pre-clinical RNA Therapeutics 21 st Global CVCT Forum – December 9-11, 2024 Title: A Randomized, Placebo-Controlled Phase 3 Study of Plozasiran in Patients with Familial Chylomicronemia Syndrome Date/Time: December 10, 2024, 7:30 p.m. EST Presenter: Jennifer Hellawell and Robert Rosenson Session: Attended Poster Viewing Session and Oral Presentation 8 th Complement-Based Drug Development Summit – December 10-11, 2024 Title: Targeting Complement C3 and Factor B with siRNA: Concept to Clinic Date/Time: December 11, 2024, 10:00 a.m. EST Presenter: James Hamilton Session: Advancing Complement Therapies: Pathways, Clinical Insights & Future Directions 22 nd World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease (WCIRDC) – December 12-14, 2024 Title: PALISADE: A Phase 3 Study to Assess the Efficacy and Safety of Plozasiran in Adults with Genetically or Clinically-Defined Familial Chylomicronemia Syndrome (FCS) at high risk of Acute Pancreatitis (AP) Date/Time: December 13, 2024, 7:30-9:00 a.m. PST Presenter: Nick Leeper and Ira Goldberg Session: Poster and Abstract Oral Presentation Session Presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website. About Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing. For more information, please visit www.arrowheadpharma.com , or follow us on X (formerly Twitter) at @ArrowheadPharma , LinkedIn , Facebook , and Instagram . To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts . 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These forward-looking statements include, but are not limited to, statements about the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; our expectations regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future; our beliefs and expectations regarding milestone, royalty or other payments that could be due to or from third parties under existing agreements; and our estimates regarding future revenues, research and development expenses, capital requirements and payments to third parties. These statements are based upon our current expectations and speak only as of the date hereof. 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We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances. Source: Arrowhead Pharmaceuticals, Inc.

The collaboration encompasses several clinical and preclinical programmes targeting rare diseases affecting the muscles, lungs and central nervous system. Credit: Stock-Asso/Shutterstock. Arrowhead Pharmaceuticals has entered a worldwide licensing and partnership agreement with Sarepta Therapeutics to develop treatments for rare genetic diseases. Arrowhead will receive an $825m payment: $500m in cash and a $325m equity investment at a 35% premium. It is also set to receive $250m, disbursed in equal instalments over five years, and qualifies for an extra $300m in forthcoming payments linked to the enrolment of certain cohorts in a Phase I/II trial. The company also stands to gain royalties on commercial sales and up to $10bn in potential future milestone payments. The collaboration encompasses clinical and preclinical programmes targeting rare diseases affecting the muscles, lungs and central nervous system (CNS). Sarepta can propose up to six new targets for which Arrowhead will perform discovery and preclinical development activities leveraging the Targeted RNAi Molecule (TRiM) platform. Sarepta will receive an exclusive licence for those programmes and handle activities related to their clinical development and commercialisation. The deal could yield development milestone payments for Arrowhead ranging from $110m to $410m, and sales milestone payments from $500m to $700m, per programme. Additionally, Arrowhead is entitled to tiered royalties on commercial sales. Sarepta’s president and CEO Doug Ingram is set to join the Arrowhead board of directors. Arrowhead is tasked with manufacturing clinical drug supply for all programmes under the licence and collaboration, as well as commercial drug products for the four programmes that are in studies at present. The completion of the transaction is anticipated in early 2025, contingent upon the expiration or termination of the waiting period mandated by the Hart-Scott-Rodino Antitrust Improvements Act of 1976, along with other standard conditions. Arrowhead Pharmaceuticals CEO and president Christopher Anzalone stated: “We estimate that this transaction extends Arrowhead’s cash runway into 2028 and potentially through multiple new drug launches, including wholly owned and partnered programmes. “We now turn our focus as a company to launching investigational plozasiran for the treatment of familial chylomicronaemia syndrome potentially in 2025, pending FDA review and approval, which would be our first commercial product.” In September 2024, the US Food and Drug Administration granted breakthrough therapy status to Arrowhead’s plozasiran.